Abu dhabi: The Department of Health - Abu Dhabi has announced the launch and funding of a UAE-first clinical trial for a novel gene therapy targeting MerTK-related retinitis pigmentosa (RP) developed by Opus Genetics. This rare inherited eye disease causes progressive vision loss and eventual blindness, with no approved treatments currently available. This groundbreaking initiative reflects Abu Dhabi's commitment to advancing innovative solutions for rare conditions and reinforces the Emirate's position as a global hub for life sciences and biomedical innovation.
According to Emirates News Agency, the first-of-its-kind clinical trial is the result of a collaborative effort between the Department of Health - Abu Dhabi and the Authority of Social Contribution - Ma'an, reflecting their shared commitment to advancing healthcare research and innovation in Abu Dhabi. The initiative brings together Opus Genetics, a US-based clinical-stage biopharmaceutical company, the Innovative Research Oversight and Support (IROS) division of the M42 group, and Cleveland Clinic Abu Dhabi to accelerate the development of novel gene therapies for Inherited Retinal Diseases, which affect approximately five percent of the population in this region.
Cleveland Clinic Abu Dhabi will serve as the clinical site, while IROS will oversee the trial design, patient recruitment, and clinical operations. Clinical development activities are expected to commence in 2026, marking a milestone for rare disease treatment in Abu Dhabi and the region. The study will evaluate the safety and efficacy of a novel AAV-based gene therapy developed by Opus Genetics for this rare condition. The therapy targets the MerTK gene, which fails to properly recycle protein in the retina when it has mutated, leading to progressive vision loss. Adeno-associated viruses (AAVs) are widely recognized as safe and effective vectors for delivering healthy genes into cells, making them a potentially powerful tool for treating inherited diseases.
Dr. Noura Al Ghaithi, Undersecretary of the Department of Health - Abu Dhabi, expressed pride in supporting the development of a novel therapy that could transform the lives of patients living with inherited blindness. Abdullah Al Ameri, Director-General of the Authority of Social Contribution - Ma'an, emphasized the importance of clinical trials in advancing healthcare and improving quality of life. George Magrath, M.D., CEO of Opus Genetics, highlighted the trial as the first real opportunity to change the course of a disease that has historically led to inevitable vision loss.
Dr. Fahed Almarzooqi, CEO of M42's Integrated Health Solutions, and Dr. Georges-Pascal Haber, CEO of Cleveland Clinic Abu Dhabi, both underscored the significance of this partnership in cementing Abu Dhabi's position as a global hub for biotech innovation and life sciences research, translating scientific breakthroughs into real-world treatments for patients with urgent, unmet needs.